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RPE65

RPE65 Overview

English Name	Retinoid isomerohydrolase	Approved Drugs	1
Drugs in Clinical Trials	5	Target Synonym	EC:5.3.3.22，Retinoid isomerohydrolase，All-trans-retinyl-palmitate hydrolase，Retinal pigment epithelium-specific 65 kDa protein，RPE65，RPE65， Retinoid Isomerohydrolase，Retinol Isomerase，Retinal Pigment Epithelium-Specific Protein 65kDa，EC 5.3.3.22，Retinoid Isomerohydrolase RPE65，Retinal Pigment Epithelium Specific Protein 65，EC 5.3.3.-，EC:3.1.1.64，Lutein Isomerase，Meso-Zeaxanthin Isomerase，BCO Family， Member 3，Retinitis Pigmentosa 20 (Autosomal Recessive)，RBP-Binding Membrane Protein，EC 3.1.1.64，MRPE65，SRPE65，BCO3，LCA2，Rd12，RP20，P63
Highest Development Stage	Approved

English Name	Retinoid isomerohydrolase
Target Synonym	EC:5.3.3.22，Retinoid isomerohydrolase，All-trans-retinyl-palmitate hydrolase，Retinal pigment epithelium-specific 65 kDa protein，RPE65，RPE65， Retinoid Isomerohydrolase，Retinol Isomerase，Retinal Pigment Epithelium-Specific Protein 65kDa，EC 5.3.3.22，Retinoid Isomerohydrolase RPE65，Retinal Pigment Epithelium Specific Protein 65，EC 5.3.3.-，EC:3.1.1.64，Lutein Isomerase，Meso-Zeaxanthin Isomerase，BCO Family， Member 3，Retinitis Pigmentosa 20 (Autosomal Recessive)，RBP-Binding Membrane Protein，EC 3.1.1.64，MRPE65，SRPE65，BCO3，LCA2，Rd12，RP20，P63
Approved Drugs	1
Drugs in Clinical Trials	5
Highest Development Stage	Approved

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RPE65 Synonyms

BCO3, LCA2, RP20, mRPE65, p63, rd12, sRPE65

Clinical Drug Information

English Name	Research Code	Highest Development Stage	Company	Indication	Clinical Trial
Emixustat Hydrochloride	ACU-02; ACU-4429; ACU-4429 HCl	Phase 3 Clinical	Acucela Inc	Anetoderma; Geographic Atrophy; Stargardt Disease; Diabetic Retinopathy; Macular Degeneration	Details
LX-101	LX-101	Phase 3 Clinical	Shanghai Langsheng Biological Technology Co Ltd	Retinal Dystrophies; Retinal Dysplasia; Retinal Degeneration	Details
HORA-RPE65 (Horama)	HORA-RPE65	Phase 2 Clinical	Horama Sa	Leber Congenital Amaurosis	Details
HG-004	HG-004	Phase 2 Clinical	HuiGene Therapeutics Ltd	Leber Congenital Amaurosis; Retinal Diseases	Details
FT-001(Frontera Therapeutics)	FT-001; FT-001(Frontera Therapeutics)	Phase 2 Clinical	Frontera Therapeutics Inc	Retinal Dysplasia	Details

Marketed Drug Information

English Name	Research Code	Highest Development Stage	Company	Earliest Brand Name	Earliest Approval Country	Earliest Indication	Earliest Approval Company	Earliest Approval Date	Indication	Clinical Trial
Voretigene neparvovec-rzyl	2SPI046IKD; rAAV.hRPE65v2; AAV2-hRPE65v2; SPKRPE-65; rAAV2-CBSB-hRPE65; AAV2.RPE65	Approved	Childrens Hospital of Philadelphia	Luxturna	United States	Retinal Dysplasia	Spark Therapeutics Inc	2017-12-19	Vision Disorders; Retinal Dystrophies; Leber Congenital Amaurosis; Retinal Dysplasia	Details

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